Pharmaceutical Research and Manufacturers of America (PhRMA) has formally introduced new Ex­panded Access principles to assist companies in evaluating whether to voluntarily grant patients access to an investigational treat­ment. Originally introduced to its members last December for review and discussion, PhRMA’s newly implemented Expanded Access principles were recently adopted with commitments from each member.

The rapid growth in compassionate care and the growing influence of patient communities has led PhRMA and the Biotechnology Industry Organization (BIO) to separately increase patient advocacy by establishing standards when member com­panies should consider granting expanded access to their investigational products.

In 2014, the FDA saw a 92% increase in “com­passionate use” from its expanded access program—the largest jump since 2010. The agency approved 1,873 emergency access requests and rejected only nine applications, according to data compiled by the Regulatory Affairs Professional Society. PhRMA’s release of new principles also is in part a response to the recently established Right to Try laws now accepted in 20 states with another 20 states pending. Although expanded access programs receive approval from the FDA and an institutional review board, under the Right to Try laws, patients and their providers are not required to obtain these approvals.

PhRMA lists a series of considerations for its member companies to follow when providing patients with access to an unap­proved investigational drug outside of a clinical trial. The principles include:

• the illness must be serious or life threatening;
• the unapproved investigational drug should be active in clinical development (in mid-to-late stage trials);
• having sufficient robust preliminary safety and efficacy data;
• significant dosing information to determine that the preliminary benefit-risk balance is positive for the specific indication for which the compassionate use request was made; and
• whether the expanded access program could compromise the conduct of clinical trials needed to obtain regulatory approval.

The FDA has its own regulatory pathway for accommodating patient requests to gain access to investigational drugs under compassionate use. The FDA governs the requests from patients and their physi­cians. However, under federal regulations, no company is required to grant access to its investigational products, as expanded access can be costly, dangerous to patients, can compromise research integrity and can potentially delay or harm drug approval. Granting expanded access is voluntary, at the discretion of each manufacturer.

“These are essentially industry voluntary commitments in a number of different areas related to clinical trials, clinical trial transpar­ency and a new section for providing inves­tigational drugs to patients in the expanded access program,” said Jeff Franker, PhRMA vice president and senior legal counsel. “We distinguish them from the FDA’s regulations on compassionate use programs as ours involve voluntary commitments by our com­panies and the FDA requirements provide a basic launching off point.”

“In addition to adopting standards, compa­nies are trying to enhance transparency about these expanded access programs, so now when a patient with a serious or life threaten­ing disease requests a drug before approval and wants to understand how to get it, the patient’s physician will have a central point of outreach at each company,” Franker added.

PhRMA has asked its member companies to establish a telephone or internet-based information source to facilitate communica­tions about expanded access programs. Patients who are eligible for expanded access to an investigational drug outside of a clinical trial should know that geographic limitations alone would generally not be considered a barrier to participation in clinical trials.

The PhRMA principles also state that beyond granting access to an investigational drug through an expanded access program, there should be no delay, interference or compromise in the completion of clinical trials that are intended to support the drug’s FDA approval. The new PhRMA principles also outline ways companies can protect research participants and ensure objectivity in research.

“It is important for the patient’s physician and the medical staff at a biopharmaceutical company to discuss whether it provides the best possible treatment option for the pa­tient before going through the FDA process,” wrote Bill Chin, M.D., PhRMA executive vice president, scientific and regulatory affairs, in a June 3 PhRMA blog posting. “Through these new principles, PhRMA affirms its commitment to working with all stakehold­ers to determine ways to help accelerate access to new medicines for the patients who most need them.”

Email comments to Ronald at ronald.rosenberg@centerwatch.com. Follow @RonRCW

This article was reprinted from Volume 19, Issue 24, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »